CA3048661A1 - Compositions et methodes d'inhibition de l'expression genique du facteur xii - Google Patents
Compositions et methodes d'inhibition de l'expression genique du facteur xii Download PDFInfo
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- CA3048661A1 CA3048661A1 CA3048661A CA3048661A CA3048661A1 CA 3048661 A1 CA3048661 A1 CA 3048661A1 CA 3048661 A CA3048661 A CA 3048661A CA 3048661 A CA3048661 A CA 3048661A CA 3048661 A1 CA3048661 A1 CA 3048661A1
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- fxii
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- antisense strand
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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/7115—Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
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- A—HUMAN NECESSITIES
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- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
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- A—HUMAN NECESSITIES
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- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/713—Double-stranded nucleic acids or oligonucleotides
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- A—HUMAN NECESSITIES
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- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
- A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
- A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
- A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
- A61P7/02—Antithrombotic agents; Anticoagulants; Platelet aggregation inhibitors
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
- A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering N.A.
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Abstract
L'invention concerne des agents d'interférence ARN permettant d'inhiber l'expression du gène de facteur XII (FXII). L'invention concerne en outre des compositions pharmaceutiques contenant un ou plusieurs agents de type ARNi de FXII conjointement avec un ou plusieurs excipients permettant l'administration du ou des agents de type ARNi à une cellule hépatique in vivo. L'administration du ou des agents de type ARNi de FXII à des cellules hépatiques in vivo permet d'inhiber l'expression du gène FXII et de traiter l'angio-dème, notamment l'angio-dème héréditaire (HAE) et la thromboembolie veineuse (VTE), ainsi que des maladies associées à l'angio-dème.
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201762451868P | 2017-01-30 | 2017-01-30 | |
US62/451,868 | 2017-01-30 | ||
PCT/US2018/015866 WO2018140920A1 (fr) | 2017-01-30 | 2018-01-30 | Compositions et méthodes d'inhibition de l'expression génique du facteur xii |
Publications (1)
Publication Number | Publication Date |
---|---|
CA3048661A1 true CA3048661A1 (fr) | 2018-08-02 |
Family
ID=62978807
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CA3048661A Pending CA3048661A1 (fr) | 2017-01-30 | 2018-01-30 | Compositions et methodes d'inhibition de l'expression genique du facteur xii |
Country Status (7)
Country | Link |
---|---|
US (1) | US20190382763A1 (fr) |
EP (1) | EP3573623A4 (fr) |
JP (2) | JP2020505415A (fr) |
CN (1) | CN110234326A (fr) |
AU (1) | AU2018213379A1 (fr) |
CA (1) | CA3048661A1 (fr) |
WO (1) | WO2018140920A1 (fr) |
Families Citing this family (18)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
CN108271386B (zh) | 2015-05-06 | 2022-07-15 | 阿尔尼拉姆医药品有限公司 | 因子XII(哈格曼因子)(F12)、激肽释放酶B、血浆(夫列契因子)1(KLKB1)和激肽原1(KNG1)iRNA组合物及其使用方法 |
KR20230115344A (ko) | 2016-09-02 | 2023-08-02 | 애로우헤드 파마슈티컬스 인코포레이티드 | 표적화 리간드 |
DK3607069T3 (da) | 2017-04-05 | 2022-11-21 | Silence Therapeutics Gmbh | Produkter og sammensætninger |
EP3649243A1 (fr) * | 2017-07-07 | 2020-05-13 | Alnylam Pharmaceuticals, Inc. | Procédés de traitement ou de prévention de maladies associées à la voie d'activation de contact à l'aide de compositions d'arni ciblant le facteur xii (facteur hageman) (f12) |
WO2019105418A1 (fr) | 2017-12-01 | 2019-06-06 | 苏州瑞博生物技术有限公司 | Oligonucléotide double brin, composition et conjugué comprenant un oligonucléotide double brin, procédé de préparation et utilisation associés |
US11660347B2 (en) | 2017-12-01 | 2023-05-30 | Suzhou Ribo Life Science Co., Ltd. | Nucleic acid, composition and conjugate containing same, preparation method, and use thereof |
CN110945130B (zh) | 2017-12-01 | 2024-04-09 | 苏州瑞博生物技术股份有限公司 | 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途 |
US11414665B2 (en) | 2017-12-01 | 2022-08-16 | Suzhou Ribo Life Science Co., Ltd. | Nucleic acid, composition and conjugate comprising the same, and preparation method and use thereof |
CN116375774A (zh) | 2017-12-29 | 2023-07-04 | 苏州瑞博生物技术股份有限公司 | 缀合物及其制备方法和用途 |
CN111655849B (zh) | 2018-08-21 | 2024-05-10 | 苏州瑞博生物技术股份有限公司 | 一种核酸、含有该核酸的药物组合物和缀合物及其用途 |
US11896674B2 (en) | 2018-09-30 | 2024-02-13 | Suzhou Ribo Life Science Co., Ltd. | SiRNA conjugate, preparation method therefor and use thereof |
CN113330117B (zh) * | 2019-01-18 | 2024-05-28 | 苏州瑞博生物技术股份有限公司 | 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途 |
WO2021153687A1 (fr) * | 2020-01-30 | 2021-08-05 | エーザイ・アール・アンド・ディー・マネジメント株式会社 | Complexe d'acide nucléique et composition pharmaceutique le contenant |
CN116670280A (zh) * | 2020-12-18 | 2023-08-29 | Ionis 制药公司 | 用于调节因子xii的化合物和方法 |
WO2022162161A1 (fr) * | 2021-01-30 | 2022-08-04 | E-Therapeutics Plc | Composés oligonucléotidiques conjugués, leurs procédés de fabrication et leurs utilisations |
AU2022215065A1 (en) * | 2021-01-30 | 2023-07-27 | E-Therapeutics Plc | Conjugated oligonucleotide compounds, methods of making and uses thereof |
TW202334413A (zh) * | 2021-08-13 | 2023-09-01 | 美商艾拉倫製藥股份有限公司 | 第十二因子(F12)iRNA組成物及其使用方法 |
WO2024062413A1 (fr) * | 2022-09-21 | 2024-03-28 | Janssen Biotech, Inc. | Nouveaux nucléosides phosphates stabilisés et leurs analogues |
Family Cites Families (3)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
EP2371857A1 (fr) * | 2010-04-01 | 2011-10-05 | CSL Behring GmbH | Inhibiteurs de facteur XII pour traiter la maladie de poumon interstitiel |
CN108064313B (zh) * | 2015-03-17 | 2021-07-30 | 箭头药业股份有限公司 | 用于抑制因子xii的基因表达的组合物和方法 |
CN108271386B (zh) * | 2015-05-06 | 2022-07-15 | 阿尔尼拉姆医药品有限公司 | 因子XII(哈格曼因子)(F12)、激肽释放酶B、血浆(夫列契因子)1(KLKB1)和激肽原1(KNG1)iRNA组合物及其使用方法 |
-
2018
- 2018-01-30 EP EP18744616.6A patent/EP3573623A4/fr active Pending
- 2018-01-30 AU AU2018213379A patent/AU2018213379A1/en not_active Abandoned
- 2018-01-30 JP JP2019541173A patent/JP2020505415A/ja active Pending
- 2018-01-30 CA CA3048661A patent/CA3048661A1/fr active Pending
- 2018-01-30 CN CN201880008973.6A patent/CN110234326A/zh active Pending
- 2018-01-30 WO PCT/US2018/015866 patent/WO2018140920A1/fr unknown
-
2019
- 2019-07-29 US US16/524,822 patent/US20190382763A1/en not_active Abandoned
-
2022
- 2022-12-02 JP JP2022193512A patent/JP2023036613A/ja active Pending
Also Published As
Publication number | Publication date |
---|---|
US20190382763A1 (en) | 2019-12-19 |
CN110234326A (zh) | 2019-09-13 |
EP3573623A1 (fr) | 2019-12-04 |
WO2018140920A1 (fr) | 2018-08-02 |
JP2020505415A (ja) | 2020-02-20 |
EP3573623A4 (fr) | 2020-11-25 |
AU2018213379A1 (en) | 2019-07-18 |
JP2023036613A (ja) | 2023-03-14 |
WO2018140920A8 (fr) | 2019-07-25 |
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